EU/3/07/486: Orphan designation for the treatment of retinitis pigmentosa

Adenovirus associated viral vector serotype 4 containing the human RPE65 gene

Table of contents

Overview

On 14 November 2007, orphan designation (EU/3/07/486) was granted by the European Commission to Centre Hospitalier Universitaire de Nantes, France, for adenovirus associated viral vector serotype 4 containing the human RPE65 gene for treatment of retinitis pigmentosa.

The sponsorship was transferred to Horama SAS, France, in May 2015.

In November 2016 Horama SAS changed name to Horama SA.

The sponsor’s name was changed from Horama SAS to Coave Therapeutics and the address was updated in August 2022.

Key facts

Active substance
Adenovirus associated viral vector serotype 4 containing the human RPE65 gene
Intended use
Treatment of retinitis pigmentosa
Orphan designation status
Positive
EU designation number
EU/3/07/486
Date of designation
14/11/2007
Sponsor

Coave Therapeutics
63 B Avenue Ledru Rollin
75012 Paris
France
E-mail: contact@coavetx.com

Documents related to this orphan designation evaluation

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.

EU register of orphan medicines

The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

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